Denali Therapeutics Q3 2024 Earnings Report
Key Takeaways
Denali Therapeutics reported its Q3 2024 financial results, highlighting progress in its Transport Vehicle (TV)-enabled portfolio, particularly the Enzyme TV (ETV) franchise with tividenofusp alfa for MPS II, and advancements in the DNL126 study for MPS IIIA.
On track to file for accelerated approval of tividenofusp alfa in MPS II in early 2025 after a successful meeting with the FDA in the third quarter.
Preliminary data from Phase 1/2 study of DNL126 in MPS IIIA demonstrate a robust reduction from baseline in CSF heparan sulfate levels, including normalization.
Study of DNL126 in MPS IIIA was expanded to support a potential accelerated path.
Preclinical publication on oligonucleotide TV (OTV) technology demonstrates broad and deep brain biodistribution of oligonucleotides.
Denali Therapeutics
Denali Therapeutics
Forward Guidance
Denali anticipates an increase of approximately 5-10% compared to 2023 cash operating expenses. This updated guidance is associated with increased activities to support filing of a BLA in early 2025 and commercial readiness for tividenofusp alfa in MPS II, and to accelerate the development of additional therapeutic programs in Denali's TV platform portfolio.
Positive Outlook
- Increased activities to support filing of a BLA in early 2025
- Commercial readiness for tividenofusp alfa in MPS II
- Accelerated development of additional therapeutic programs in Denali's TV platform portfolio
- Advancing OTV:MAPT, targeting tau for Alzheimer’s disease, and OTV:SNCA, targeting alpha-synuclein for Parkinson’s disease, in the investigational new drug (IND)-enabling stage of development.
- Plans to advance a TfR-targeting ATV:Abeta molecule as well as a CD98hc-targeting ATV:Abeta molecule into development for Alzheimer's disease.